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Hemophilia Therapeutics Pipeline Analysis

P&S Market Research-Hemophilia Therapeutics Pipeline Analysis report

Hemophilia Therapeutics Pipeline Analysis, 2017 - Clinical Trials & Results, Patent, Designation, Collaboration, and Other Developments

Published: June 2017
Report Code: LS10934
Available Format: pdf
Pages: 116

Disease Overview

Hemophilia is a genetic disorder characterized by excessive bleeding and delayed blood clotting. There are two types of hemophilia, hemophilia A (also known as classic hemophilia) and hemophilia B. According to the Centers for Disease Control and Prevention, the incidence of hemophilia stands at, one out of every 5,000 male births and as many as 20,000 males in the U.S., living with either of the two types of hemophilia, presently. The prevalence of hemophilia A is around four times high as compared to the prevalence of hemophilia B. It has been observed that the male population is more commonly affected by the disease, as compared to the female population, since hemophilia is inherited in an X-linked recessive genetic pattern, where females are generally carriers of the disease. Hemophilia A results from a deficiency of clotting Factor VIII, whereas hemophilia B (also known as the Christmas disease) is caused by a deficiency of clotting Factor IX, both of these are essential for the proper functioning of blood clotting. The cause of the deficiency of clotting factors is thought to be a mutation in one of the genes that codes for the synthesis of these clotting factor, which are required to form a blood clot.

Pipeline Analysis

Significant growth is expected in the therapeutic pipeline of hemophilia, mainly attributed to increasing collaboration between pharma as well as biotech companies, educational institutes and associations for the development of hemophilia therapeutics. Increasing prevalence of hemophilia and technological advancements are some of the major factors for the development of novel therapies for treatment of hemophilia. For instance, PolyXen is an enabling platform technology for protein drug delivery. Zinc finger nuclease mediated in vivo genome editing approach makes use of the endogenous albumin gene locus, a highly expressing and liver-specific site that can be edited with ZFNs to accept and express therapeutic genes.  Positive clinical results of Phase III and Phase II drug candidates with lesser side effects are supporting the development of hemophilia therapeutics. Many awareness programs organized by pharma as well as biotechnology companies are also adding to the development of hemophilia therapeutics pipeline.

 

NUMBER OF HEMOPHILIA DRUG CANDIDATES UNDER DEVELOPMENT (2017)

Hemophilia Therapeutics Pipeline Analysis

As of June 2017, the hemophilia pipeline comprises of approximately 53 drug candidates, of which one drug candidate has been filed by LFB SA as biologics license application, four drug candidates are in Phase III stage, four drug candidates are in Phase II stage, 11 drug candidates are in Phase I, 27 drug candidates are in Pre-Clinical stage and six drug candidates are in the Discovery stage of development.

Competitive Landscape

Many pharmaceutical companies are developing hemophilia drug candidates that are present in different phases of development. For instance, Novo Nordisk A/S, Chugai Pharmaceutical Co. Ltd., F. Hoffmann-La Roche AG, Bayer AG, Merck Sharp & Dohme Corp., Pfizer Inc., Oncolytics Biotech Inc. and others have their hemophilia drug candidates in different stages of development.

Apart from the players mentioned above, some of the other key players developing drugs for the treatment of hemophilia include, Sangamo BioSciences, Inc., Caisson Biotech, Inc., XL-protein GmbH, mAbxience SA, Prothix BV, Immusoft Corporation, 3B Pharmaceuticals GmbH, GeneOne Life Science, Inc., Sernova Corp., Arcturus Therapeutics, Inc., REGiMMUNE Corporation, Alteogen, Inc., Glycotope GmbH.

Scope for Customization

P&S Market Research offers customization as per specific business requirements of clients. Illustrative customization within the scope of this report includes:

  • Market Forecast – Market analysis and forecast for the drug candidates in the latest stage of development
  • Company Profiles – Wider company coverage in terms of detailed analysis or additional company profiles

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